Breakthrough Drug Designation May Be Misleading

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Breakthrough Drug Designation May Be Misleading
Breakthrough Drug Designation May Be Misleading

HealthDay News — Use of the word "breakthrough" in the U.S. Food and Drug Administration's expedited approval process may be misleading, according to a research letter published in the Journal of the American Medical Association.

The U.S. Congress in 2012 gave FDA the power to designate a drug as a "breakthrough therapy" if preliminary clinical evidence suggests an advantage over existing medications. But a survey of 692 doctors revealed that many tended to misinterpret "breakthrough." Doctors often believed the drugs were supported by stronger evidence than the law requires to achieve that designation, lead author Aaron Kesselheim, MD, JD, MPH, a faculty member at Brigham and Women's Hospital in Boston, told HealthDay.

In the survey, 9 out of 10 doctors chose one hypothetical drug over another identical one solely based on the "breakthrough" designation, even though both were taken on the same schedule, had similar side effects, and were equally covered by the patient's insurance. In addition, 52% incorrectly believed that strong evidence from randomized trials is needed to earn the breakthrough designation. The survey also revealed a widespread lack of understanding about the meaning of FDA approval for a new drug, the researchers reported.

The FDA released the following statement in response to the study: "In general, the FDA does not comment on specific studies, but evaluates them as part of the body of evidence to further our understanding about a particular issue and assist in our mission to protect public health."


Kesselheim AS, Woloshin S, Eddings W, Franklin JM, Ross KM, Schwartz LM. Physicians' Knowledge About FDA Approval Standards and Perceptions of the “Breakthrough Therapy” Designation. JAMA. 2016;315(14):1516-1518. doi:10.1001/jama.2015.16984.

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